Cell Therapeutics Shares Crash as Lymphoma Drug Gets Shot Down by FDA Panel

3/22/10Follow @xconomy

[Updated: 4:35 pm Eastern] Cell Therapeutics’ new lymphoma drug failed to win a recommendation today from a panel of cancer experts who advise the FDA. Shares of the company fell by 48 percent on the news.

The crash came after FDA’s Oncologic Drugs Advisory Committee voted 9 to 0 today against the Seattle-based company’s application to start marketing pixantrone as a treatment for patients with relapsed forms of non-Hodgkin’s lymphoma whose disease has worsened after two prior rounds of therapy. The FDA isn’t required to follow the advice of its expert advisory panels, although it often does. The agency’s deadline to complete its review of the drug is April 23.

The vote is a major setback for Cell Therapeutics (NASDAQ: CTIC). The company has no marketed products, and pixantrone represents its only chance of a candidate that could generate U.S. sales anytime soon. Cell Therapeutics had $37.8 million in cash and investments heading into this year, which isn’t enough to operate the business through the end of September.

The chair of the FDA panel, Gail Eckhardt of the University of Colorado at Denver, said the Cell Therapeutics application was “disturbing,” partly because it only enrolled 140 of the 320 patients needed to generate a statistically valid result.

“I don’t see this as a well designed or well executed study. The evidence speaks against it,” said Wyndham Wilson, a lymphoma expert at the National Cancer Institute, and a member of the panel. Wilson also made an earlier point that pixantrone is a modified member of a chemotherapy class known as anthracyclines which are already known to be active treatments that are widely used in the U.S. and Europe. Most of the patients who responded in the Cell Therapeutics trial were in less developed countries, who have fewer treatment options.

The company didn’t appear to have any more luck persuading the regulators at the FDA. The chief of the FDA’s cancer drug division, Richard Pazdur, started his presentation out this morning by emphasizing that the Cell Therapeutics application was based on a “single incomplete trial.” He pointed out that the data from the 140 patient study is comparable to what the FDA usually sees from the second of the three phases of clinical trials usually required to win approval. FDA staff suggested that the company might be better off running other trials of pixantrone in combination with other chemotherapy regimens. The panel didn’t appear to disagree with that.

“It’s an interesting agent. I’d encourage the sponsor to develop it further, but it’s way too early now,” Wilson said, as he explained his ‘No’ vote.

The company didn’t say immediately say what it plans to do next, but it did offer a statement that suggested it hasn’t given up on pixantrone.

“We continue to believe that pixantrone should be considered as a treatment option for patients with relapsed/refractory aggressive non-Hodgkin’s lymphoma. There is a significant medical need to bring safe and effective therapies to this very sick patient population,” Bianco said in a statement. “We are committed to working closely with the FDA to address the committee’s comments as quickly as we can.”

[Updated: 4:35 pm, with closing stock price.] Shares of Cell Therapeutics lost almost half their value, falling to 47 cents, at the close of trading today after the FDA panel’s recommendation.

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